Duchenne Muscular Dystrophy
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CLINICAL_TRIALS trial_result · 2017-08-18Clinical Trial Update: A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy [Phase 1]
Clinical trial update reporting completion of a Phase 1 pharmacokinetic study of oral deflazacort in children and adolescents with Duchenne …
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CLINICAL_TRIALS trial_result · 2017-07-18Clinical Trial Update: A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects [Phase 1]
A Phase 1, double-blind, escalating-dose, randomized, placebo-controlled study in non-ambulant Duchenne muscular dystrophy patients evaluati…
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CLINICAL_TRIALS trial_result · 2017-04-11Clinical Trial Update: Virtual Reality in Individuals With Duchenne Muscular Dystrophy [NA]
A clinical trial evaluating virtual reality for individuals with Duchenne Muscular Dystrophy has completed.
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CLINICAL_TRIALS research · 2017-04-11Clinical Trial Update: Analysis of a Virtual Reality Task in Patients With Duchenne Muscular Dystrophy [NA]
Clinical trial update analyzing a virtual reality task in patients with Duchenne Muscular Dystrophy.
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CLINICAL_TRIALS trial_result · 2017-03-23Clinical Trial Update: Open Label Study of GSK2402968 in Subjects With Duchenne Muscular Dystrophy [Phase 3]
Open-label Phase 3 trial of GSK2402968 for Duchenne muscular dystrophy has been terminated.
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CLINICAL_TRIALS trial_result · 2017-03-21Clinical Trial Update: Cardiomyopathy in DMD: Lisinopril vs. Losartan [NA]
This entry notes a completed clinical trial comparing lisinopril versus losartan for cardiomyopathy in Duchenne muscular dystrophy, with res…
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CLINICAL_TRIALS trial_result · 2017-03-01Clinical Trial Update: A Study of Tadalafil for Duchenne Muscular Dystrophy [Phase 3]
Phase 3 trial for tadalafil in Duchenne muscular dystrophy was terminated; results posted in 2017.
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FDA approval · 2017-02-22FDA Approved: EXONDYS 51 — Supplemental Application (Sarepta Therapeutics, Inc.)
FDA approves a supplemental application for Exondys 51 (eteplirsen) from Sarepta Therapeutics.
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FDA approval · 2017-02-09FDA Approved: EMFLAZA — Original Application (PTC Therapeutics, Inc.)
The FDA approved EMFLAZA (deflazacort) from PTC Therapeutics via original NDA for the treatment of Duchenne muscular dystrophy in patients a…
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FDA approval · 2017-02-09FDA Approved: EMFLAZA — Original Application (PTC Therapeutics, Inc.)
FDA approved EMFLAZA (deflazacort) in an original application from PTC Therapeutics for Duchenne muscular dystrophy.
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CLINICAL_TRIALS trial_result · 2017-01-18Clinical Trial Update: This is a Study to Get More Information About Non Ambulatory Boys & Men With Duchenne Muscular Dystrophy
A completed clinical trial aimed at gathering more information about non-ambulatory boys and men with Duchenne muscular dystrophy.
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CLINICAL_TRIALS trial_result · 2016-11-07Clinical Trial Update: A Phase I/II, Open Label, Escalating Dose, Pilot Study to Assess Effect, Safety, Tolerability and PK of Multiple SC Doses of Drisapersen in Patients With Duchenne Muscular Dystrophy and to Assess the Potential for IV Dosing as an Alternative Route of Administration [Phase 2]
Phase I/II open-label escalating-dose pilot study of subcutaneous Drisapersen in Duchenne muscular dystrophy to assess safety, tolerability,…
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CLINICAL_TRIALS trial_result · 2016-10-14Clinical Trial Update: Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy [Phase 2]
Phase 2 clinical trial of ACE-031 in Duchenne muscular dystrophy has been terminated with results posted in 2016.
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CLINICAL_TRIALS trial_result · 2016-10-04Clinical Trial Update: Myocardial Involvement in Carriers of Duchenne Muscular Dystrophy: An MRI-study
This completed MRI study examined myocardial involvement in carriers of Duchenne muscular dystrophy. It aimed to understand potential heart …
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FDA approval · 2016-09-19FDA Approved: EXONDYS 51 — Original Application (Sarepta Therapeutics, Inc.)
FDA approved Exondys 51 (eteplirsen) from Sarepta Therapeutics as the original NDA for Duchenne muscular dystrophy amenable to exon 51 skipp…
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EMA withdrawal · 2016-09-06EMA Application withdrawn: Kyndrisa — BioMarin International Limited
The EMA withdrew the application for Kyndrisa for Duchenne muscular dystrophy.
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CLINICAL_TRIALS research · 2016-08-17Clinical Trial Update: Research of Biomarkers in Duchenne Muscular Dystrophy Patients
This is an update on a clinical trial investigating biomarkers in Duchenne Muscular Dystrophy patients; the study (NCT01380964) sponsored by…
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CLINICAL_TRIALS trial_result · 2016-08-11Clinical Trial Update: A Device for Rapid, Painless, Bedside Muscle Evaluation of Children
This clinical trial update reports the completion of a study evaluating a rapid, painless bedside device for assessing muscle function in ch…
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CLINICAL_TRIALS research · 2016-04-21Clinical Trial Update: Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)
This entry provides an update on a longitudinal natural history study of Duchenne Muscular Dystrophy.
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CLINICAL_TRIALS research · 2016-04-12Clinical Trial Update: Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
This clinical trial update describes an observational study of Duchenne Muscular Dystrophy patients who could theoretically be treated with …
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CLINICAL_TRIALS trial_result · 2016-03-24Clinical Trial Update: Electrical Impedance Myography and Ultrasound as Biomarkers of Duchenne Muscular Dystrophy
A completed study evaluating electrical impedance myography and ultrasound as potential biomarkers for Duchenne Muscular Dystrophy.
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CLINICAL_TRIALS trial_result · 2016-02-03Clinical Trial Update: Mother-caregivers of Children With Duchenne Muscular Dystrophy [NA]
This entry reports on a completed clinical trial focusing on mother-caregivers of children with Duchenne Muscular Dystrophy. The sponsor is …
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CLINICAL_TRIALS trial_result · 2016-01-28Clinical Trial Update: Ramipril Versus Carvedilol in Duchenne and Becker Patients [Phase 4]
Phase 4 clinical trial comparing Ramipril versus Carvedilol in Duchenne muscular dystrophy and Becker muscular dystrophy patients; current s…
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CLINICAL_TRIALS research · 2015-11-25Clinical Trial Update: The Safety, Efficacy and Tolerability of Remote Ischemic Preconditioning as a Therapy to DMD [NA]
This entry reports on a clinical trial evaluating remote ischemic preconditioning as a therapy for Duchenne muscular dystrophy, with a focus…
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CLINICAL_TRIALS trial_result · 2015-11-09Clinical Trial Update: Efficacy Study of AVI-4658 to Induce Dystrophin Expression in Selected Duchenne Muscular Dystrophy Patients [Phase 2]
Phase 2 trial evaluating AVI-4658 to induce dystrophin expression in Duchenne Muscular Dystrophy patients has completed with results posted …